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골수 이형성 증후군(MDS) : 시장 인사이트, 역학 및 시장 예측(2036년)

Myelodysplastic Syndrome - Market Insight, Epidemiology, and Market Forecast - 2036

발행일: | 리서치사: 구분자 DelveInsight | 페이지 정보: 영문 200 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




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골수 이형성 증후군(MDS)에 대한 인사이트와 동향

  • DelveInsight의 분석에 따르면, 2025년 주요 시장(미국, EU4(독일, 프랑스, 이탈리아, 스페인), 영국 및 일본)의 MDS 시장 규모는 약 43억 달러인 것으로 나타났습니다.
  • Blood Cancer United에 따르면, MDS 환자의 약 25-30%가 고위험형을 보입니다. 증례의 약 30%에서는 고위험형 MDS나 이차성 급성 골수성 백혈병(AML)으로 진행될 가능성이 있습니다.
  • MDS UK에 따르면, MDS는 희귀질환입니다. 영국에서는 인구 10만 명당 연간 약 4건의 발생률을 보이고 있습니다. MDS가 발병하는 환자의 전형적인 연령은 70세 전후입니다. 50세 미만의 MDS 환자는 10% 미만입니다.
  • MDS는 주로 고령자에게 발병하며, 진단 당시의 연령 중앙값은 약 71세입니다. 표준화 순생존율은 1년 시점에서 약 83%, 5년 시점에서 51%로 추정됩니다.
  • MDS 시장에서 사업을 전개하고 있는 제약 회사로는 브리스톨-마이어스 스퀴브(REBLOZYL), 제론(RYTELO), 세르비에 파마슈티컬스(TIBSOVO) 등이 있습니다.
  • 베네토클락스는 고위험 골수 이형성 증후군(HR-MDS) 분야에서 가장 영향력 있는 신흥 치료법 중 하나가 될 것으로 기대되고 있습니다. 이는 급성 골수성 백혈병(AML)에 대한 풍부한 임상 경험과 저메틸화제와의 병용 요법에 관한 유망한 데이터에 의해 뒷받침되고 있습니다.
  • 루스파텔셉트-aamt(REBLOZYL)은 적혈구 수혈이 필요할 가능성이 있는 매우 낮은 위험도에서 중간 위험도의 MDS를 가진 성인 환자 중, 지금까지 적혈구 조혈 자극제(ESA)를 사용한 적이 없는(ESA 미치료) 경우의 빈혈에 대한 1차 치료의 표준 요법입니다.
  • MDS의 파이프라인은 매우 탄탄하며, 베네토클락스(VENCLEXTA)(애브비), 벡마릴리맙(팔론 파마슈티컬스), ASTX727(타이호 종양), 오르카-T(오르카 바이오), R289(리겔 파마슈티컬스), 리사프트클락스(APG-2575)(어센티지 파마), 오르타시데닙(리겔 파마슈티컬스), 오필노플라스트(HT-6184)(하리아 테라퓨틱스) 등이 안전하고 효과적인 치료법으로 개발이 진행되고 있습니다.
  • Halia Therapeutics사는 EHA 2026에서 LR-MDS를 대상으로 한 오필노플라스트의 2상 임상시험 최종 결과를 발표할 예정입니다. 채택된 초록에 따르면, 본 임상시험은 IWG 2018 기준에 따른 혈액학적 개선(HI)이라는 주요 평가 지표를 달성하였으며, 이는 해당 환자 집단에서 오필노플라스트의 치료 가능성을 뒷받침하는 결과입니다.
  • 위험도 기반 치료 전략의 도입으로 MDS의 치료 환경은 완전히 달라졌습니다. 저메틸화제는 오랫동안 치료의 주축이 되어 왔지만, 환자의 최대 50%는 이러한 치료로부터 거의 또는 전혀 혜택을 받지 못했습니다.

본 MDS 시장 보고서에서는 표준 치료, 임상 실무, 진화하는 치료 알고리즘 등 현재 시장 상황에 대해 종합적인 분석을 제시하고 있습니다. 또한, MDS 환자의 부담 추이, 수익 및 시장 점유율 추이, 피크 시기의 환자 점유율 및 치료 도입 현황에 대한 분석을 평가함과 동시에, 세계 각 지역 시장 규모에 대한 상세한 평가 및 성장률 예측(과거 데이터 및 2022년-2036년 예측)을 제공합니다. 본 보고서에서는 MDS 분야의 주요 미충족 요구 사항을 부각시키고, 경쟁 구도 및 임상 현황을 분석하여 고부가가치의 성장 기회를 도출함으로써, 향후 시장 성장 가능성에 대한 명확한 전망을 제시하고 있습니다.

골수 이형성 증후군(MDS) 시장을 주도하는 주요 요인

고령화의 진행과 질병 부담 증가

MDS는 주로 고령자에게 발병하며, 65세 이상 인구에서 발병률이 현저히 증가하고 있습니다. 주요 시장의 고령 인구 증가에 따라 MDS의 진단 및 치료 건수가 증가할 것으로 예상되며, 이에 따라 시장 기회가 확대될 것으로 전망됩니다.

병용 요법의 활용 확대

저메틸화제, B세포 림프종-2(Bcl-2) 억제제, 면역조절요법 및 신규 표적 치료제를 이용한 병용요법이 점점 더 높이 평가받고 있습니다. 이러한 전략을 통해 얻은 긍정적인 임상 결과가 치료법 도입을 촉진하고 시장 성장을 이끌 것으로 예측됩니다.

임상시험 활동의 활성화와 파이프라인의 혁신

세포 치료, 면역 치료, 표적 치료제를 특징으로 하는 탄탄한 파이프라인은 MDS에 대한 업계의 높은 관심을 반영하고 있습니다. 새로운 치료법에 대한 지속적인 임상 개발과 승인 가능성으로 인해 치료 선택지가 다양해지고, 시장 확대가 촉진될 것으로 예측됩니다.

골수 이형성 증후군(MDS)의 이해와 치료 알고리즘

골수 이형성 증후군(MDS)의 개요와 진단

MDS는 조혈 기능 부전을 특징으로 하며, 그 결과 정상적인 혈액 세포의 생성이 불충분해지는 드물고 이질성이 높은 골수 질환군입니다. 이 질환은 조혈모세포의 유전적 및 후성유전적 이상에 기인하며, 지속적인 혈구 감소, 골수이형성 및 급성 골수성 백혈병(AML)으로 진행될 위험 증가를 초래합니다. MDS는 주로 고령자에게 발병하며, 만성 빈혈, 재발성 감염, 출혈성 합병증, 삶의 질 저하, 그리고 지속적인 의료 관리가 필요하다는 점으로 인해 큰 임상적 부담을 초래합니다. MDS의 생물학적 및 임상적 이질성으로 인해 위험도에 따른 치료 접근법이 개발되어 왔으나, 여전히 새로운 질환 수정 요법의 필요성이 대두되고 있습니다.

MDS의 진단은 임상 평가, 검사 소견, 골수 검사, 그리고 세포유전학적 또는 분자생물학적 분석을 종합하여 이루어집니다. 환자는 일반적으로 전혈구계수(CBC)를 통해 확인되는 원인을 알 수 없는 지속적인 혈구 감소 증상을 보입니다. 골수 천자 및 생검은 이형성, 블라스트 비율, 세포 밀도를 평가하는 데 필수적이며, 한편 세포유전학적 검사 및 차세대 염기서열 분석(NGS)은 진단, 위험도 분류 및 치료법 선택을 뒷받침하는 염색체 이상과 유전자 변이를 확인하는 데 도움이 됩니다. 예후는 일반적으로 IPSS-R이나 IPSS-M과 같은 점수 산정 시스템을 통해 평가됩니다.

골수 이형성 증후군(MDS)의 현재 치료 현황

MDS의 치료 전략은 질환의 위험도, 증상의 중증도, 연령 및 환자의 전신 상태를 바탕으로 결정됩니다. 저위험군 환자의 경우, 빈혈에 대해 적혈구 수혈, 적혈구 조혈 자극 인자(ESA), 그리고 루스파텔셉트를 포함한 지지 요법이 시행되는 경우가 많습니다. 고위험군 환자에게는 일반적으로 아자시티딘이나 데시타빈과 같은 저메틸화제를 이용한 질환 수정 요법이 시행됩니다. 또한, 적격한 환자의 경우, 유일한 근치적 치료법이 될 수 있는 동종 조혈모세포 이식이 시행되는 경우가 있습니다. Bcl-2 억제제, 면역 표적 치료제, 새로운 세포 치료법 등 새로운 치료법들이 MDS 치료의 현 상황을 한층 더 변화시킬 것으로 기대되고 있습니다.

골수 이형성 증후군(MDS)의 역학

MDS의 역학 분석 및 예측에 관한 주요 조사 결과

  • 2025년 주요 7개국에서 MDS의 총 발병자 수는 약 4만 3,000명이었습니다. 이 수치는 2036년까지 증가할 것으로 예측됩니다.
  • LR-MDS로 분류되는 환자의 대부분은 증상을 동반한 빈혈을 보이며, 이는 전체 증례의 약 89%에서 관찰됩니다.
  • EU4 및 영국 중에서 MDS 신규 환자 수가 가장 많았던 나라는 독일이었으며, 그 다음은 프랑스였습니다. 한편, 2025년 기준으로 MDS 신규 환자 수가 가장 적었던 나라는 스페인이었습니다.
  • 미국에서는 MDS 사례의 대부분이 RAEB/MDS-EB 아형이며, 2025년에는 약 7,000건을 차지했습니다.
  • 분석에 따르면, 2025년 독일에서 SF3B1 변이를 특징으로 하는 사례가 MDS 중 가장 많았으며, 약 900건을 차지했습니다.

골수 이형성 증후군(MDS) 시장 전망

MDS의 치료 환경은 기존의 대증 요법이나 저메틸화제(HMA)에서 특정 분자 이상이나 질환의 생물학적 특성에 대응하도록 설계된 표적 치료, 적혈구 성숙 촉진제, 그리고 병용 요법으로 큰 변화를 겪고 있습니다. 현재 이 시장은 루스파텔셉트-aamt, 이메텔스타트, 이보시데닙 등 승인된 치료제들에 의해 지탱되고 있으며, 이들 각각은 서로 다른 환자층을 차지하고 있습니다. 루스파텔셉트는 저위험 수혈 의존성 빈혈에 대한 1차 치료제로서의 입지를 확고히 하고 있는 반면, 이메텔스타트는 ESA 내성 환자에서 지속적인 수혈 불필요 상태를 실현함으로써 경쟁력을 높이고 있습니다. 이보시데닙은 IDH1 변이를 가진 환자로 제한되지만, MDS에서 바이오마커 기반 치료 접근법의 역할이 확대되고 있음을 보여줍니다. 이메텔스타트는 2026년 1분기에 5,180만 달러의 순매출을 기록했으며, 이는 2025년 4분기 대비 8% 증가한 수치로, 출시 초기 단계부터 견조한 시장 침투세를 보이고 있습니다. 한편, 루스파텔셉트는 적응증 범위가 넓고 시장에 조기에 진출한 덕분에, 여전히 시장에서 매우 큰 입지를 유지하고 있습니다.

신흥 치료법 중에서도 베네토클락스는 HR-MDS 분야에서 가장 영향력 있는 파이프라인 자산 중 하나가 될 것으로 기대되고 있습니다. 이 약물은 이미 급성 골수성 백혈병(AML) 분야에서 확고한 임상적 입지를 다졌으며, 현재 진행 중인 골수 이형성 증후군(MDS) 임상시험에서는 아자시티딘과의 병용 요법을 통해 유망한 반응률이 나타나고 있습니다. 진행 중인 임상시험에서 생존 이점이 입증되고, 안전성이 허용 범위 내에 있음이 확인된다면, 베크마릴리맙은 재발성·난치성 MDS 환자에서 보여준 유망한 효능으로 인해 주목을 받고 있습니다. 초기 임상 데이터에 따르면, 지속적인 치료 효과와 양호한 내약성이 확인되었으며, 이는 치료 선택지가 제한적인 환자 집단에서 이 약물의 잠재적 역할을 뒷받침하고 있습니다. 또 다른 주목할 만한 신흥 자산은 리사프트크라크스로, 병용 요법에서 유망한 효능의 징후가 보고되어 차세대 BCL-2 억제제로 자리매김하고 있습니다. 이 약물의 향후 경쟁력은 안전성, 투여의 유연성, 혹은 효능의 지속성 측면에서 베네토클락스에 비해 우위를 보여줄 수 있느냐에 달려 있습니다.

  • 리겔 파마슈티컬스는 파이프라인에 알타시데닙과 R289를 보유하고 있으며, MDS 시장에서 유력한 경쟁 기업으로 부상하고 있습니다. 이 회사가 표적 치료와 면역 조절 치료 두 분야 모두에서 입지를 다지고 있다는 점은 경쟁력을 강화할 뿐만 아니라, MDS 환자의 여러 하위 그룹을 대상으로 치료할 기회를 제공합니다.
  • 베크마릴리맙은 재발성·난치성(r/r) 질환을 앓고 있으며 치료 선택지가 제한된 환자를 대상으로 하는 차별화된 면역요법 후보로 자리매김하고 있습니다. 후기 임상시험에서 긍정적인 결과가 도출된다면, 치료 옵션이 여전히 제한적인 미충족 의료 수요가 높은 분야에서 이 치료법의 도입이 촉진될 가능성이 있습니다.
  • 텔로메라제 억제제: 이메텔스타트는 수혈 의존성 빈혈을 동반한 국소성 MDS(LR-MDS) 환자에 대해 승인된 최초의 텔로메라제 억제제입니다. 이 약물군은 텔로메라아제 활성을 억제함으로써 악성 조혈 전구세포를 표적으로 삼습니다. 이러한 승인을 통해, 지지 요법이나 적혈구 조혈 자극제를 뛰어넘는 새로운 질환 수정 치료 접근법이 도입되었습니다.
  • Bcl-2 억제제: 베네토클락스와 리사프트클락스는 악성 조혈세포의 아포토시스를 촉진하는 Bcl-2 억제제로 분류됩니다. 베네토크라크스를 기반으로 한 병용 요법은 고위험 MDS(HR-MDS) 분야에서 큰 관심을 받고 있는 반면, 리사프트크라크스는 독자적인 유효성 및 안전성 프로파일을 제공할 가능성을 지닌 차세대 Bcl-2 억제제로서 개발이 진행되고 있습니다.
  • 세포 치료: 오르카-T는 동종 세포 치료에 속하며, 조혈모세포 이식 후의 치료 결과를 최적화하도록 설계되었습니다. 기존의 약물 요법과 달리, 이 접근법은 이식 성공률을 높이는 동시에 이식편 대 숙주 질환(GVHD) 등의 합병증을 완화하는 것을 목표로 하며, 적격 환자를 대상으로 한 고도로 전문화된 치료 전략입니다.

MDS 분야의 혁신은 더 이상 단일 치료 기전에 의해 주도되는 것이 아니라, 적아구 성숙 촉진제, 텔로머라제 억제제, 표적 분자 치료, 세포 사멸 유도제, 면역 조절제 및 세포 치료법의 융합을 통해 추진되고 있습니다. 이러한 다양화로 인해 치료 알고리즘이 재구축되고, 분자적 및 임상적 특성에 기반한 환자 계층화가 진행되며, 예측 기간 동안 지속적인 시장 성장이 촉진될 것으로 기대됩니다.

자주 묻는 질문

  • 2025년 골수 이형성 증후군(MDS) 시장 규모는 어떻게 예상되나요?
  • 골수 이형성 증후군(MDS) 환자의 고위험형 비율은 얼마인가요?
  • 골수 이형성 증후군(MDS)의 전형적인 발병 연령은 어떻게 되나요?
  • MDS 치료에 사용되는 주요 약물은 무엇인가요?
  • MDS의 치료 환경은 어떻게 변화하고 있나요?
  • MDS의 진단 방법은 무엇인가요?
  • MDS의 역학적 분석 결과는 어떤가요?

목차

제1장 주요 인사이트

제2장 서론

제3장 골수 이형성 증후군 : 주요 요약

제4장 주요 이벤트

제5장 골수 이형성 증후군 : 역학 및 시장 조사 방법

제6장 골수 이형성 증후군 : 시장 개요

제7장 골수 이형성 증후군 : 질환 배경과 개요

제8장 골수 이형성 증후군 : 역학 및 환자 인구

제9장 골수 이형성 증후군 : 환자 경과

제10장 골수 이형성 증후군 : 시판 치료제

제11장 골수 이형성 증후군 : 신흥 치료법

제12장 골수 이형성 증후군 : 주요 7개국 분석

제13장 골수 이형성 증후군 : 미충족 요구

제14장 골수 이형성 증후군 : SWOT 분석

제15장 골수 이형성 증후군 : KOL(Key Opinion Leader)의 견해

제16장 시장 참여 및 상환

제17장 부록

제18장 DelveInsight의 서비스 내용

제19장 면책사항

제20장 DelveInsight에 대해

LSH 26.07.27

Myelodysplastic Syndrome (MDS) Insights and Trends

  • According to DelveInsight's analysis, the MDS market size was found to be ~USD 4,300 million in the leading markets (the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan) in 2025.
  • As per Blood Cancer United, approximately 25-30% of MDS patients have high-risk disease. The disease can progress to high-risk MDS and secondary AML in approximately 30% of cases.
  • As per MDS UK, MDS is a rare disease. In the UK, there are about 4 cases per 100,000 population per year. The typical age at which patients develop MDS is around 70 years old. Fewer than 10% of MDS patients are younger than 50 years.
  • MDS primarily affects older adults, with a median age at diagnosis of approximately 71 years. The standardized net survival is estimated to be about 83% at 1 year and 51% at 5 years.
  • The pharmaceutical companies active in the MDS market include Bristol Myers Squibb (REBLOZYL), Geron (RYTELO), Servier Pharmaceuticals (TIBSOVO), and others.
  • Venetoclax is expected to be one of the most influential emerging therapies in HR-MDS. Backed by extensive clinical experience in AML and encouraging combination data with hypomethylating agents.
  • Luspatercept-aamt (REBLOZYL) is a standard of care for the first-line treatment of anemia without previous erythropoiesis stimulating agent use (ESA-naive) in adult patients with very low- to intermediate-risk MDS who may require RBC transfusions.
  • The pipeline of MDS is very robust, emerging therapies such as Venetoclax (VENCLEXTA) (AbbVie), bexmarilimab (Faron Pharmaceuticals), ASTX727 (Taiho Oncology), Orca-T (Orca Bio), R289 (Rigel Pharmaceuticals), lisaftoclax (APG-2575) (Ascentage Pharma), olutasidenib (Rigel Pharmaceuticals), ofirnoflast (HT-6184) (Halia Therapeutics), and others are being developed for safe and efficacious treatments.
  • Halia Therapeutics is scheduled to present final Phase II results for ofirnoflast in LR-MDS at EHA 2026. The accepted abstract indicated that the study achieved its primary endpoint of hematological improvement (HI) per IWG 2018 criteria, supporting the therapeutic potential of ofirnoflast in this patient population.
  • The introduction of risk-adapted treatment strategies has transformed the MDS treatment landscape. While hypomethylating agents long served as the cornerstone of therapy, up to 50% of patients derived little or no benefit from these treatments.

DelveInsight's 'Myelodysplastic Syndrome (MDS) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of the MDS, historical and forecasted epidemiology, as well as the MDS market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

The MDS market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates MDS patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in MDS and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

Key Factors Driving the Myelodysplastic Syndrome (MDS) Market

Increasing Aging Population and Disease Burden

MDS predominantly affects older adults, with incidence rising significantly in individuals aged 65 years and above. The growing elderly population across major markets is expected to increase the number of diagnosed and treated MDS cases, thereby expanding the market opportunity.

Expanding Use of Combination Therapies

Combination approaches involving hypomethylating agents, B-cell lymphoma-2 (Bcl-2) inhibitor, immune-modulating therapies, and novel targeted agents are being increasingly evaluated. Positive clinical outcomes from these strategies are expected to drive treatment adoption and market growth.

Growing Clinical Trial Activity and Pipeline Innovation

A robust pipeline featuring cell therapies, immunotherapies, and targeted agents reflects strong industry interest in MDS. Continued clinical development and potential approvals of novel therapies are expected to diversify treatment options and stimulate market expansion.

Myelodysplastic Syndrome (MDS) Understanding and Treatment Algorithm

Myelodysplastic Syndrome (MDS) Overview and Diagnosis

MDS is a group of rare, heterogeneous bone marrow disorders characterized by ineffective hematopoiesis, leading to insufficient production of healthy blood cells. The disease arises from genetic and epigenetic abnormalities in hematopoietic stem cells, resulting in persistent cytopenias, bone marrow dysplasia, and an increased risk of progression to acute myeloid leukemia (AML). MDS primarily affects older adults and presents a substantial clinical burden due to chronic anemia, recurrent infections, bleeding complications, reduced quality of life, and the need for ongoing medical management. The biological and clinical heterogeneity of MDS has led to the development of risk-adapted treatment approaches and continues to drive the need for novel disease-modifying therapies.

The diagnosis of MDS is based on a combination of clinical evaluation, laboratory testing, bone marrow examination, and cytogenetic or molecular analyses. Patients typically present with persistent unexplained cytopenias identified through complete blood counts (CBCs). Bone marrow aspiration and biopsy are essential for assessing dysplasia, blast percentage, and cellularity, while cytogenetic testing and next-generation sequencing (NGS) help identify chromosomal abnormalities and gene mutations that support diagnosis, risk stratification, and treatment selection. Prognosis is commonly assessed using scoring systems such as the IPSS-R and IPSS-M.

Current Myelodysplastic Syndrome (MDS) Treatment Landscape

Treatment strategies for MDS are guided by disease risk, symptom burden, age, and patient fitness. Lower-risk patients are often managed with supportive care, including red blood cell transfusions, erythropoiesis-stimulating agents (ESAs), and luspatercept for anemia. Higher-risk patients generally receive disease-modifying therapies such as hypomethylating agents, including azacitidine and decitabine, while eligible patients may undergo allogeneic hematopoietic stem cell transplantation, the only potentially curative treatment. Emerging therapies such as Bcl-2 inhibitors, immune-targeted agents, and novel cellular therapies are expected to further transform the MDS treatment landscape.

Myelodysplastic Syndrome (MDS) Unmet Needs

The section "unmet needs of MDS" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.

1. Poor understanding of bone marrow failure mechanisms independent of blast burden

2. Lack of effective therapies after hma failure

3. Limited therapies addressing disease progression

4. Persistent transfusion dependence, and others.....

Myelodysplastic Syndrome (MDS) Epidemiology

Key Findings from MDS Epidemiological Analysis and Forecast

  • The total incident population of MDS in the 7MM were ~43,000 in 2025. These numbers are expected to increase by 2036.
  • Patients classified as LR-MDS, frequently present with symptomatic anemia, occurring in approximately 89% of cases.
  • Among the EU4 and the UK, Germany had highest incident cases of MDS, followed by France. On the other hand, Spain had the lowest incident population of MDS, in 2025.
  • In the US, the majority of MDS cases were of the RAEB/MDS-EB subtype accounting for nearly 7,000 cases in 2025.
  • As per the analysis, mutation-specific cases of SF3B1 mutation accounted for the highest number of cases in MDS, i.e., ~900 cases, in Germany in 2025.

Myelodysplastic Syndrome (MDS) Drug Analysis & Competitive Landscape

The MDS drug chapter provides a detailed, market-focused review of the emerging pipeline across Phase III/I clinical trials and preclinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the MDS treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the MDS therapeutics market.

Approved Therapies for Myelodysplastic Syndrome (MDS)

Imetelstat (RYTELO): Geron

Imetelstat is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Imetelstat is the first and only telomerase inhibitor approved by the US FDA and the European Commission. It has US market exclusivity till June 2029 and till March 2035 in EU. It also has orphan drug exclusivity till June 2031 in US and till March 2025 in EU. RYTELO is positioned to lead in second-line Lower-Risk Myelodysplastic Syndrome (LR-MDS).

Ivosidenib (TIBSOVO): Servier

Ivosidenib is a oral, targeted, first-in-class precision medicine developed by Servier that blocks the isocitrate dehydrogenase-1 (IDH1) mutation. It is primarily prescribed for adults with relapsed/refractory (r/r) or newly diagnosed MDS. The FDA approval of this indication is supported by a pivotal Phase I, open-label study in IDH1-mutated R/R MDS patients. The therapy has demonstrated clinical benefit in hematologic cancers and targeted oncology settings driven by IDH1 mutations.

Myelodysplastic Syndrome (MDS) Pipeline Analysis

Venetoclax (VENCLEXTA): AbbVie

Venetoclax is a selective Bcl-2 inhibitor. The US FDA granted a Breakthrough Therapy Designation (BTD) to venetoclax in combination with azacitidine for the treatment of adult patients with previously untreated intermediate-, high- and very high-risk MDS. Venetoclax is a prescription medicine used in chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). Venetoclax is being developed by AbbVie and Roche. It is jointly commercialized by Genentech in the US and by AbbVie outside of the US.

Orca-T: OrcaBio

Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment MDS. Orca Bio is evaluating Orca-T across multiple clinical trials in MDS, including Phase III, Phase II, and Phase I/Ib studies assessing different donor settings, conditioning regimens, and transplant approaches. By improving transplant outcomes and lowering transplant-related complications, Orca-T has the potential to expand access to curative HSCT for patients with HR-MDS.

Myelodysplastic Syndrome (MDS) Key Players, Market Leaders and Emerging Companies

  • Geron
  • Servier
  • Bristol Myers Squibb
  • AbbVie
  • OrcaBio
  • Ascentage Pharma
  • Faron Pharmaceuticals
  • Taiho Oncology
  • Rigel Pharmaceuticals, and others

Myelodysplastic Syndrome (MDS) Drug Updates

  • Geron in its 2026 corporate presenation mentions PTE application is filed in the US and EU for RYTELO patents and RYTELO MOU (MDS) patent respectively.
  • In May 2026, Halia Therapeutics announced final Phase II results for ofirnoflast (HT-6184) in patients with LR-MDS. The data will be presented in an oral session at the European Hematology Association (EHA) 2026 Hybrid Congress.
  • In May 2026, Geron Corporation announced that the first real-world evidence study of imetelstat in patients with LR-MDS will be presented at the European Hematology Association (EHA) 2026 Congress.
  • In May 2026, Faron Pharmaceuticals announces that an abstract for its bexmarilimab Phase I/II study has been accepted as a poster presentation at the European Hematology Association (EHA) 2026 Congress. The BEXMAB study investigates bexmarilimab in combination with standard of care (SoC) in MDS.
  • In March 2026, Faron Pharmaceuticals announcesd that investigators at City of Hope are in the process of developing a Phase II investigator-initiated clinical trial (IIT) in collaboration with Faron to evaluate bexmarilimab in participants with relapsed or refractory MDS.
  • In Feburary 2026, Orca Bio announced new clinical data presented at the 2026 Tandem Meetings of ASTCT and CIBMTR. In Phase III Precision-T study, Orca-T demonstrated superior OS and RFS with reduced non-relapse mortality in patients with myelodysplastic syndromes compared to PTCy-based transplants.
  • In Feburary 2026, Orca Bio announced first patients dosed in the Phase II SERENE-T study evaluating Orca-T with RIC in MDS.
  • In December 2025, Faron Pharmaceuticals announced that two abstracts featuring data from the Phase I/II bexmarilimab study have been accepted for presentation at the American Society of Hematology (ASH) 2025 Annual Meeting. The Phase I/II BEXMAB study assessed impact of bexmarilimab plus azacitidine in patients with treatment-naive HR-MDS.
  • In December 2025, Rigel Pharmaceuticals announced updated data from its ongoing Phase Ib study evaluating R2891 in patients with R/R LR-MDS. R289 continued to be generally well tolerated and at doses of >=500 mg QD preliminary efficacy was observed in elderly.

Myelodysplastic Syndrome (MDS) Market Outlook

The treatment landscape for MDS is undergoing a significant transformation from traditional supportive care and hypomethylating agents (HMAs) toward targeted therapies, erythroid maturation agents, and combination regimens designed to address specific molecular abnormalities and disease biology. The market is currently anchored by approved therapies such as luspatercept-aamt, imetelstat, and ivosidenib, each occupying distinct patient segments. Luspatercept has established itself as a preferred option for lower-risk transfusion-dependent anemia, while imetelstat has strengthened competition by offering durable transfusion independence in ESA-refractory patients. ivosidenib, although limited to IDH1-mutated disease, exemplifies the growing role of biomarker-driven treatment approaches in MDS. Imetelstat generated net revenue of USD 51.8 million in Q1 2026, representing an 8% increase over Q4 2025, indicating strong early commercial uptake. While luspatercept continues to maintain a substantially larger market presence owing to its broader label and earlier market entry.

Among emerging therapies, venetoclax is expected to be one of the most influential pipeline assets in the HR-MDS setting. The drug has already established a strong clinical foundation in AML, and ongoing MDS studies have shown encouraging response rates when combined with azacitidine. If ongoing trials demonstrate a survival benefit and manageable safety profile. Bexmarilimab has attracted attention due to its encouraging activity in patients with r/r MDS. Early clinical data have demonstrated durable responses and a favorable tolerability profile, supporting its potential role in a patient population with limited therapeutic options. Another notable emerging asset is lisaftoclax, which has reported promising efficacy signals in combination regimens and is being positioned as a next-generation BCL-2 inhibitor. Its future competitiveness will depend on whether it can demonstrate advantages over venetoclax in terms of safety, dosing flexibility, or durability of response.

  • Rigel Pharmaceuticals is emerging as a strong competitor in the MDS market with olutasidenib and R289 in its pipeline. The company's presence across both targeted and immune-modulating therapies strengthens its competitive position and provides opportunities to address multiple MDS patient subgroups.
  • Bexmarilimab has positioned itself as a differentiated immunotherapy candidate targeting patients with r/r disease and limited treatment options. Positive late-stage data could support uptake in a high-unmet-need setting where therapeutic alternatives remain limited.

Drug Class/Insights into Leading Emerging and Marketed Therapies in MDS (2022-2036 Forecast)

The MDS market (2022-2036 forecast) is evolving from a landscape historically dominated by supportive care and hypomethylating agents toward a more targeted and biomarker-driven treatment paradigm. Advances in disease biology have led to the development of therapies addressing ineffective hematopoiesis.

  • Telomerase Inhibitors: Imetelstat represents the first telomerase inhibitor approved for LR-MDS patients with transfusion-dependent anemia. This class targets malignant hematopoietic progenitor cells by inhibiting telomerase activity. It's approval has introduced a novel disease-modifying approach beyond supportive care and erythropoiesis-stimulating agents.
  • Bcl-2 Inhibitors: Venetoclax and lisaftoclax belong to the Bcl-2 inhibitor class, which promotes apoptosis of malignant hematopoietic cells. Venetoclax-based combinations have generated considerable interest in HR-MDS, while lisaftoclax is being developed as a next-generation Bcl-2 inhibitor with the potential to offer differentiated efficacy and safety profiles.
  • Cell Therapy: Orca-T belongs to the allogeneic cell therapy class and is designed to optimize outcomes following hematopoietic stem cell transplantation. Unlike conventional pharmacological therapies, this approach seeks to improve transplant success while reducing complications such as graft-versus-host disease, representing a highly specialized therapeutic strategy for eligible patients.

MDS innovation is no longer driven by a single therapeutic mechanism but rather by a convergence of erythroid maturation agents, telomerase inhibitors, targeted molecular therapies, apoptosis-inducing agents, immune modulators, and cellular therapies. This diversification is expected to reshape treatment algorithms, increase patient stratification based on molecular and clinical characteristics, and drive sustained market growth throughout the forecast period.

Myelodysplastic Syndrome (MDS) Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the MDS drug's uptake, performance at peak, factors affecting performance during prime years of growth, patient uptake by therapy, and anticipated sales generated by each drug.

Among approved therapies, luspatercept is expected to maintain strong uptake in LR-MDS patients with anemia, supported by its established efficacy in reducing transfusion burden and its expanding role in earlier lines of treatment. Imetelstat is anticipated to witness increasing adoption among transfusion-dependent LR-MDS patients following ESA failure, driven by its novel telomerase inhibition mechanism and durable transfusion independence outcomes. Meanwhile, Ivosidenib is expected to experience more selective uptake due to its applicability to the relatively small subset of patients harboring IDH1 mutations, although its precision-medicine approach supports strong value within this niche population.

The emerging MDS pipeline is expected to drive increased competition across both lower-risk and higher-risk disease segments. Venetoclax is expected to achieve strong uptake in HR-MDS, supported by its established clinical use in AML and promising combination data with hypomethylating agents. Lisaftoclax (APG-2575) may emerge as a competitive next-generation Bcl-2 inhibitor if it demonstrates advantages in efficacy or tolerability over existing Bcl-2-based regimens. Bexmarilimab is likely to gain traction in R/R MDS, where unmet need remains high, while ofirnoflast and R289 may see selective adoption if their novel immune and inflammatory pathway-targeting approaches translate into meaningful clinical benefit.

Detailed insights of emerging therapies' drug uptake is included in the report

Market Access and Reimbursement of Approved therapies in Myelodysplastic Syndrome (MDS)

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Reimbursement is a crucial factor that affects the drug's access to the market. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, many payment models are being considered by payers and other industry insiders.

NOTE: Further Details are provided in the final report....

Myelodysplastic Syndrome (MDS) Therapies Price Scenario & Trends

Pricing and analogue assessment of MDS therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, closest and most appropriate analogue selection for emerging therapies, and understanding of how pricing influences market access, adherence, and long-term uptake.

Industry Experts and Physician Views for Myelodysplastic Syndrome (MDS)

To keep up with MDS market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the MDS emerging therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in MDS, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.

DelveInsight's analysts connected with 15+ KOLs to gather insights at the country level. Centers such as Stanford MDS Center, Memorial Sloan Kettering Cancer Center, and University Medical Center Mainz etc. were contacted. Their opinion helps understand and validate current and emerging MDS therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in MDS.

Qualitative Analysis: SWOT and Conjoint Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis.

In the SWOT analysis of MDS, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint analysis analyzes emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

The team of analysts analyzes promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are majorly observed. In addition, the scoring is also based on the route of administration, order of entry, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Scope of the Report:

  • The report covers a segment of key events, an executive summary, a descriptive overview of MDS, explaining their causes, signs and symptoms, pathogenesis, and currently available treatments.
  • Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
  • Additionally, an all-inclusive account of both the current and emerging treatments, along with the elaborative profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
  • A detailed review of the MDS market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
  • The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM MDS market.

Report Insights

  • Myelodysplastic Syndrome (MDS) Patient Population Forecast
  • Myelodysplastic Syndrome (MDS) Therapeutics Market Size
  • Myelodysplastic Syndrome (MDS) Pipeline Analysis
  • Myelodysplastic Syndrome (MDS) Market Size and Trends
  • Myelodysplastic Syndrome (MDS) Market Opportunity (Current and Forecasted)

Report Key Strengths

  • Epidemiology-based (Epi-based) Bottom-up Forecasting
  • Artificial Intelligence (AI)-Enabled Market Research Report
  • 11-Year Forecast
  • Myelodysplastic Syndrome (MDS) Market Outlook (North America, Europe, Asia-Pacific)
  • Patient Burden Trends (By Geography)
  • Myelodysplastic Syndrome (MDS) Treatment Addressable Market (TAM)
  • Myelodysplastic Syndrome (MDS) Competitive Landscape
  • Myelodysplastic Syndrome (MDS) Major Companies Insights
  • Myelodysplastic Syndrome (MDS) Price Trends and Analogue Assessment
  • Myelodysplastic Syndrome (MDS) Therapies Drug Adoption/Uptake
  • Myelodysplastic Syndrome (MDS) Therapies Peak Patient Share Analysis

Report Assessment

  • Myelodysplastic Syndrome (MDS) Current Treatment Practices
  • Myelodysplastic Syndrome (MDS) Unmet Needs
  • Myelodysplastic Syndrome (MDS) Clinical Development Analysis
  • Myelodysplastic Syndrome (MDS) Emerging Drugs Product Profiles
  • Myelodysplastic Syndrome (MDS) Market attractiveness
  • Myelodysplastic Syndrome (MDS) Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

Market Insights

  • What was the MDS market size, the market size by therapies, market share (%) distribution in 2025, and what would it look like by 2036? What are the contributing factors for this growth?
  • What are the anticipated pricing variations among different geographies for the emerging therapies in the future?
  • What can be the future treatment paradigm of MDS?
  • What are the disease risks, burdens, and unmet needs of MDS? What will be the growth opportunities across the 7MM concerning the patient population with MDS?
  • Who is the major future competitor in the market, and how will the competitors affect their market share?
  • What are the current options for the treatment of MDS? What are the current guidelines for treating MDS in the US, Europe, and Japan?

Reasons to Buy:

  • The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the MDS market.
  • Bottom up forecasting builds from the affected population to product forecasts, delivering a robust, data driven approach ideal for new therapies and novel classes.
  • Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
  • Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
  • Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
  • Detailed analysis and ranking of class-wise potential emerging therapies under the conjoint analysis section to provide visibility around leading classes.
  • To understand KOLs' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
  • Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
  • This Artificial Intelligence (AI) enabled report summarize and simplify complex datasets with in the report into clear, actionable insights for stakeholders, investors, and healthcare providers, enabling faster, data driven decisions.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary of Myelodysplastic Syndrome (MDS)

4. Key Events

  • 4.1. Upcoming Key Catalyst
  • 4.2. Key Conferences And Meetings
  • 4.3. Key Transactions And Collaborations
  • 4.4. News Flow

5. Epidemiology and Market Methodology of Myelodysplastic Syndrome (MDS)

6. Myelodysplastic Syndrome (MDS) Market Overview at a Glance

  • 6.1. Emerging Landscape Analysis (By Molecule Type, Phase, and Route of Administration [ROA])
  • 6.2. Market Share of Myelodysplastic Syndrome (MDS) By Therapies (%) in the 7MM in 2025
  • 6.3. Market Share of Myelodysplastic Syndrome (MDS) By Therapies (%) in the 7MM in 2036

7. Disease Background And Overview of Myelodysplastic Syndrome (MDS)

  • 7.1. Introduction
  • 7.2. Causes
  • 7.3. Signs And Symptoms
  • 7.4. Diagnosis
    • 7.4.1. Differential Diagnosis
    • 7.4.2. Diagnostic Algorithm
    • 7.4.3. Diagnostic Guidelines
  • 7.5. Treatment and Management
    • 7.5.1. Treatment Algorithm
    • 7.5.2. Treatment Guidelines

8. Epidemiology and Patient Population of Myelodysplastic Syndrome (MDS)

  • 8.1. Key Findings
  • 8.2. Assumption and Rationale
  • 8.3. Myelodysplastic Syndrome (MDS) Total Incident Cases in the 7MM
  • 8.4. The United States
    • 8.4.1. Myelodysplastic Syndrome (MDS) Total Incident Cases in the United States
    • 8.4.2. Myelodysplastic Syndrome (MDS) Age-specific Incident Cases in the United States
    • 8.4.3. Myelodysplastic Syndrome (MDS) Subtype-specific Incident Cases in the United States
    • 8.4.4. Myelodysplastic Syndrome (MDS) Risk-specific Incident Cases in the United States
    • 8.4.5. Myelodysplastic Syndrome (MDS) Mutation-specific Incident Cases in the United States
  • 8.5. EU4 and the UK
    • 8.5.1. Myelodysplastic Syndrome (MDS) Total Incident Cases in EU4 and the UK
    • 8.5.4. Myelodysplastic Syndrome (MDS) Age-specific Incident Cases in EU4 and the UK
    • 8.5.5. Myelodysplastic Syndrome (MDS) Subtype-specific Incident Cases in EU4 and the UK
    • 8.5.6. Myelodysplastic Syndrome (MDS) Risk-specific Incident Cases in EU4 and the UK
    • 8.5.4. Myelodysplastic Syndrome (MDS) Mutation-specific Incident Cases in EU4 and the UK
  • 8.6. Japan
    • 8.6.1. Myelodysplastic Syndrome (MDS) Total Incident Cases in Japan
    • 8.6.2. Myelodysplastic Syndrome (MDS) Age-specific Incident Cases in Japan
    • 8.6.5. Myelodysplastic Syndrome (MDS) Subtype-specific Incident Cases in Japan
    • 8.6.6. Myelodysplastic Syndrome (MDS) Risk-specific Incident Cases in Japan
    • 8.6.5. Myelodysplastic Syndrome (MDS) Mutation-specific Incident Cases in Japan

9. Patient Journey of Myelodysplastic Syndrome (MDS)

10. Marketed Therapies of Myelodysplastic Syndrome (MDS)

  • 10.1. Marketed Competitive Landscape of Myelodysplastic Syndrome (MDS)
  • 10.2. Imetelstat (RYTELO): Geron
    • 10.2.1. Product Description
    • 10.2.2. Regulatory Milestones
    • 10.2.3. Other Developmental Activities
    • 10.2.4. Summary of Pivotal Trials
    • 10.2.5. Analyst Views
  • 10.3. Ivosidenib (TIBSOVO): Servier
    • 10.3.1. Product Description
    • 10.3.2. Regulatory Milestones
    • 10.3.3. Other Developmental Activities
    • 10.3.4. Summary of Pivotal Trials
    • 10.3.5. Analyst Views

11. Emerging Therapies of Myelodysplastic Syndrome (MDS)

  • 11.1. Emerging Competitive Landscape of Myelodysplastic Syndrome (MDS)
  • 11.2. Venetoclax (VENCLEXTA): AbbVie
    • 11.2.1. Product Description
    • 11.2.2. Other Developmental Activities
    • 11.2.3. Clinical Development
      • 11.2.3.1. Clinical Trial Information
    • 11.2.4. Safety and Efficacy
    • 11.2.5. Analyst Views
  • 11.3. Orca-T: OrcaBio
    • 11.3.1. Product Description
    • 11.3.2. Other Developmental Activities
    • 11.3.3. Clinical Development
      • 11.3.3.1. Clinical Trial Information
    • 11.3.4. Safety and Efficacy
    • 11.3.5. Analyst Views

12. Myelodysplastic Syndrome (MDS): Seven Major Market Analysis

  • 12.1. Key Findings
  • 12.2. Market Outlook of Myelodysplastic Syndrome (MDS)
  • 12.3. Conjoint Analysis of Myelodysplastic Syndrome (MDS)
  • 12.4. Key Market Forecast Assumptions
    • 12.4.1. Cost Assumptions
    • 12.4.2. Pricing Trends
    • 12.4.3. Analogue Assessment
    • 12.4.4. Launch Year and Therapy Uptakes
  • 12.5. Total Market Size of Myelodysplastic Syndrome (MDS) in the 7MM
  • 12.6. The United States
    • 12.6.1. Total Market Size of Myelodysplastic Syndrome (MDS) in the United States
    • 12.6.2. Market Size of Myelodysplastic Syndrome (MDS) by Therapies in the United States
  • 12.7. EU4 and the UK
    • 12.7.1. Total Market Size of Myelodysplastic Syndrome (MDS) in EU4 and the UK
    • 12.7.2. Market Size of Myelodysplastic Syndrome (MDS) by Therapies in EU4 and the UK
  • 12.8. Japan
    • 12.8.1. Total Market Size of Myelodysplastic Syndrome (MDS) in Japan
    • 12.8.2. Market Size of Myelodysplastic Syndrome (MDS) by Therapies in Japan

13. Unmet Needs of Myelodysplastic Syndrome (MDS)

14. SWOT Analysis of Myelodysplastic Syndrome (MDS)

15. KOL Views of Myelodysplastic Syndrome (MDS)

  • 15.1. Expert/KOL Interview Highlights

16. Market Access and Reimbursement

  • 16.1. The US
  • 16.2. In EU4 and the UK
    • 16.2.1. Germany
    • 16.2.2. France
    • 16.2.3. Italy
    • 16.2.4. Spain
    • 16.2.5. United Kingdom
  • 16.3. Japan
  • 16.4. Summary and Comparison of Market Access and Pricing Policy Developments in 2025
  • 16.5. Market Access and Reimbursement of Myelodysplastic Syndrome (MDS) Therapies

17. Appendix

  • 17.1. Bibliography
  • 17.2. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

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